Gene therapy strategies have exploited lenti or retro viral approaches for long-term gene replacement, however, their clinical applications remain limited because of potential for viral-associated oncogenesis. Recently, gene therapy strategies have attempted to use hybrid Adenovirus/Adeno-associated viruses (Ad/AAV) to combine the capacity, tropism and ease of production of adenovirus (Ad) with adeno-associated virus (AAVs) ability for site-specific integration (SSI). Although the AAV Rep78 protein is required for SSI, it also has an inhibitory effect on Ad replication, particularly when co-expressed within the Ad backbone leading difficulty in generating and integrating transgene within the back-bone of a single hybrid virus.
Researchers at Stony Brook University have identified a genetic element within the Rep gene sequence, which is inhibitory to Ad replication. This AAV Rep gene was then genetically recoded using synonymous codon pair re-engineering to overcome Reps inhibitory effects on Ad replication. The novel construct thus created provides a unique opportunity to place all genetic elements in one virus for the purpose of safely integrating a transgene into a specific region of the human genome.
Combines the advantages of Ad (high titer, high infectivity and large capacity) and integration capability of AAV Offers one-step packaging systems for rAAV viral production.
New Gene therapy vectors Improved rAAV production methods.
Wadie Bahou, Professor of Medicine, Medicine
Patrick Hearing, Professor, Molecular Genetics and Microbiology
Varsha Sitaraman, Graduate Student,
Licensing,Commercial partner,Development partner,Seeking investment
Available for License. Stony Brook University seek to develop and commercialize, by an exclusive or non-exclusive license agreement and/or sponsored research, with a company active in the area.
Sean Boykevisch, Director, Intellectual Property Partners, firstname.lastname@example.org, 6316326952
Patent application submitted
Pending US Utility covering compositions and methods of use